Ceregene Reports Data From Parkinson's Disease Phase 2b Study

SAN DIEGO, April 19, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. The trial did not demonstrate statistically significant efficacy on the primary endpoint (UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off score), as defined and prespecified in the Statistical Analysis Plan, did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE-120 and the dosing methods employed. A marked placebo effect was observed in this trial in that both the sham-surgery-control patients and the CERE-120 treated patients showed significant improvement following their surgery.

Fifty-one (51) patients with moderately advanced Parkinson's disease who could not be satisfactorily controlled with conventional Parkinson's medication were enrolled in the study at 11 leading clinical sites throughout the U.S. Approximately half of the patients received CERE-120 while the other half received sham (placebo) surgery as a control. Patients were monitored for 15-24 months to assess safety and changes in Parkinson's disease symptoms, using multiple endpoints such as the Unified Parkinson's Disease Rating Scale (UPDRS), Daily Diaries that assess motor function throughout the day, and PDQ-39 (a measure of quality of life), among others. Ceregene continues to analyze the data from this trial to gain as much information as possible.

Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc. stated, "We are disappointed that we did not achieve broader statistical significance in this small clinical trial, perhaps due in part to the marked placebo effect noted above. That said, we at Ceregene want to acknowledge the courage of all of the patients enrolled in this very important study and their family members. We also would like to acknowledge the Michael J. Fox Foundation for Parkinson's Research for their long-term support as we continued the development of this novel neurotrophic factor-based treatment with the potential to improve symptoms and also slow disease progression."

Raymond T. Bartus, Ph.D., executive vice president and chief scientific officer of Ceregene stated: "While we did not achieve the degree of efficacy we had hoped for in this trial, we are proud that our efforts have helped to establish that gene therapy can provide the enabling technology to safely deliver stable, long-term bioactive protein to targeted sites deep in the human brain and that the Parkinson's disease brain is able to show a positive response to neurotrophic factor stimulation. Hopefully, the information and insight we achieved and shared with the biomedical community will aid in the continuing effort to develop more effective therapies for many of these tragic and dehumanizing neurodegenerative diseases."

C. Warren Olanow, M.D., Chairman Emeritus of the Department of Neurology and Professor of Neuroscience at the Mount Sinai School of Medicine in New York and a clinical advisor to Ceregene noted, "This was an extremely well-conceived and designed study. It is unfortunate for patients that broader benefits from this extremely promising therapy could not be demonstrated in this clinical trial. These results illustrate how difficult it is to establish clinical efficacy with entirely novel therapeutic approaches in complicated neurological diseases. The Ceregene team, its scientific advisors and the participating clinical sites are to be congratulated for that effort and the flawless execution of this difficult scientific study."

Ceregene established a leadership position in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. A total of over 100 patients have been safely dosed in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease (ALS). Ceregene is currently looking at strategic alternatives to advance its AAV gene therapy platform.

About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of a harmless adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring function. Neurturin is a member of the same protein family as glial cell line-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 is delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen, as well as the cell bodies for these same neurons, located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, controlled and highly targeted neurturin expression for years following a single injection, confirmed in both animal and human studies.

About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.

About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development of nervous system growth factors (neurotrophic factors) as treatments for neurodegenerative and retinal disorders using gene transfer for their delivery. The company has established a leadership position in the fields of gene therapy and neurotrophic factors, having launched 6 separate clinical trials in Parkinson's and Alzheimer's disease, enrolling a total of nearly 200 patients, over 100 of whom have been administered the gene therapy products, some several years ago, with no safety serious issues. Additionally, the company has published 2 dozen peer-reviewed scientific papers describing novel nonclinical and clinical findings. Ceregene's clinical program for Alzheimer's disease involves CERE-110, an AAV2-based vector expressing nerve growth factor (NGF). A fully enrolled multi-center, controlled Phase 2 study with CERE-110 is ongoing, conducted in collaboration with the Alzheimer's Disease Cooperative Study and partially funded by a grant from the National Institutes of Health (NIH). Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology Partners and BioSante Pharmaceuticals (Nasdaq: BPAX).

About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.

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